EU/3/12/973: Orphan designation for the treatment of mucopolysaccharidosis type VII (Sly syndrome)

Recombinant human beta-glucuronidase (vestronidase alfa)


On 21 March 2012, orphan designation (EU/3/12/973) was granted by the European Commission to NDA Regulatory Science Ltd, United Kingdom, for recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome).

The sponsorship was transferred to NDA Group AB, Sweden, in December 2013 and subsequently to Ultragenyx UK Limited, United Kingdom, in May 2015.

Recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome) has been authorised in the EU as Mepsevii since 23 August 2018.

This medicine is now known as vestronidase alfa.

The sponsor’s address was updated in November 2020.

Key facts

Active substance
Recombinant human beta-glucuronidase (vestronidase alfa)
Medicine name
Intended use
Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Orphan designation status
EU designation number
Date of designation

Ultragenyx Germany GmbH
Rahel-Hirsch-Strasse 10
10557 Berlin
Tel. +4930590083651

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Mepsevii at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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